Interim analysis of a post-authorization safety study of pitolisant in treating narcolepsy: A real-world European study

被引：0

作者：

Plazzi, Giuseppe ^{[1
]}

Mayer, Geert ^{[2
]}

Bodenschatz, Ralf ^{[3
]}

Bonanni, Enrica ^{[4
]}

Cicolin, Alessandro ^{[5
]}

Della Marca, Giacomo ^{[6
]}

Dolso, Pierluigi ^{[7
]}

Strambi, Luigi Ferini ^{[8
]}

Ferri, Raffaele ^{[9
]}

Geisler, Peter ^{[10
]}

Happe, Svenja ^{[11
]}

Heidbreder, Anna ^{[12
]}

Herold, Juergen ^{[13
]}

Kallweit, Ulf ^{[14
]}

Leclair-Visonneau, Laurene ^{[15
]}

Lederer, Katharina ^{[16
]}

Liguori, Claudio ^{[17
,18
]}

Meurling, Johan ^{[19
]}

Parrino, Liborio ^{[20
]}

Proserpio, Paola ^{[21
]}

Puligheddu, Monica ^{[22
]}

Salva, Maria Antonia Quera ^{[23
]}

Remi, Jan ^{[24
]}

Romigi, Andrea ^{[25
]}

Rupprecht, Sven ^{[26
]}

Savarese, Maria Antonietta ^{[27
]}

Schaff, Jean-Luc ^{[28
]}

Terzaghi, Michele ^{[29
]}

Winter, Yaroslav ^{[30
,31
]}

Causse, Christian ^{[32
]}

Collin, Irene ^{[32
]}

Lecomte, Isabelle ^{[32
]}

Dauvilliers, Yves ^{[33
]}

机构：

[1] Azienda USL Bologna Osped Bellaria, Via Altura 3, I-40139 Bologna, Italy

[2] Hephata Klin, Schalfzentrums, Schimmelpfengstr 6, D-34613 Schwalmstadt, Germany

[3] TU Chemnitz, Carolastr 2, D-09111 Chemnitz, Germany

[4] Ctr Sonno Azienda Osped Univ Pisana Neurol, Via Roma 67, I-56100 Pisa, Italy

[5] Univ Torino, Via Cherasco 15, I-10126 Turin, Italy

[6] Fdn Policlin Univ A Gemelli Roma, Largo Gemelli 8, I-00168 Rome, Italy

[7] Univ Hosp Udine, ASUFC Santa Maria Misericordia, Dept Cardiothorac Surg, Pzzale Santa Maria Della Misericordia 15, Udine, Italy

[8] Ospidale San Raffaele Turro, Via Stamira D'Ancona 20, I-20127 Milan, Italy

[9] Oasi Res Inst IRCCS, Sleep Res Ctr, Via Conte Ruggero 73, I-94018 Troina, Italy

[10] Bezirksklinikum Regensburg, Klin Psychiat, Univ Str 84, D-93042 Regensburg, Germany

[11] Klin Maria Frieden Telgte, Krankenhaus 1, Telgte, Germany

[12] Johannes Kepler Univ Linz, Kepler Univ Hosp GmbH, JKU Dept Ophthalmol, Albert Schweitzer Campus 1,Bldg A1 Gebaude A1, A-4040 Munster, Austria

[13] Ambulantes Schaflabor Furth, Bahnhofpl 6, D-90762 Furth, Germany

[14] Witten Herdecke Univ, Dept Psychol, Alfred Herrhausen Str 50, D-58448 Witten, Germany

[15] CHU Nantes Hop Laennec, Blvd Prof Jacques Monod, F-44093 Nantes, France

[16] Adv Sleep Res GmbH, Luisenstr 54-55, D-10117 Berlin, Germany

[17] Univ Roma Tor Vergata, Dept Syst Med, Neurol Unit, Viale Oxford 81, I-00133 Rome, Italy

[18] Univ Roma Tor Vergata, Dept Syst Med, Viale Oxford 81, Rome, Italy

[19] Guys Hosp, London SE1 9RT, England

[20] Azienda Osped Univ Parma, Ctr Interdipartamentale Med Sonno, Via Gramsci 14, I-43100 Parma, Italy

[21] ASST Grande Osped Metropolitano Niguarda, Ctr Med Sonno, Dipartimento Neurosci, Piazza Osped Maggiore, I-20162 Milan, Italy

[22] Univ Cagliari, Sleep Disorder Ctr, Dept Med Sci & Publ Hlth, SS554 Bivio Sestu, Monserrato, CA, Italy

[23] Hop Raymond Poincare, SleepUnit, 74 Blvd Raymond Poincare, F-92380 Garches, France

[24] Univ Munich, Dept Neurol, Marchioninistr 15, D-81377 Munich, Germany

[25] IRCCS Neuromed, IRCCS Neuromed Ist Neurol Mediterraneo Pozzilli It, Via Atinense 18, IT-86077 Pozzilli, IS, Italy

[26] Univ Klinikum Jena, Klinikum 1, D-07747 Jena, Germany

[27] UOC Neurol Univ Francomichele Puca, Ctr Disturbi Sonno, Ctr Studio Disturbi Sonno, Piazza Giulio Cesare 11, I-70124 Bari, Italy

[28] CHRU Nancy, 29 Ave Marechal de Lattre de Tassigny, F-54035 Nancy, France

[29] Serv Neurofisiopatol IRCCS, Ctr Multidisciplinare Med Sonno, Via Mondino 2, I-27100 Pavia, Italy

[30] Johannes Gutenberg Univ Mainz, Mainz Comprehens Epilepsy & Sleep Med Ctr, Dept Neurol, Univ Med Ctr, Langenbeckstr 1, D-55131 Mainz, Germany

[31] Philipps Univ Marburg, Dept Neurol, Biegenstr 10, D-35037 Marburg, Germany

[32] Bioprojet, Paris, France

[33] CHRU Gui Chauliac, 80 Ave Augustin Fliche, F-34295 Montpellier, France

来源：

SLEEP MEDICINE | 2025年 / 129卷

关键词：

Narcolepsy; Pitolisant; Safety; Efficacy; Europe; DOUBLE-BLIND; CATAPLEXY; EFFICACY; PLACEBO; DEPRESSION; VALIDITY;

D O I：

10.1016/j.sleep.2025.02.012

中图分类号：

R74 [神经病学与精神病学];

学科分类号：

摘要：

Background: Narcolepsy is a chronic sleep disorder characterized mainly by excessive daytime sleepiness (EDS) and cataplexy in the case of narcolepsy type 1 (NT1). Pitolisant is a histamine 3 receptor antagonist/inverse agonist that reduces EDS and cataplexy in patients with narcolepsy. Methods: We performed a prospective 5-year follow-up, non-interventional study of adults with NT1 and NT2 receiving pitolisant. The primary objectives were to collect information on the long-term safety of pitolisant and analyze the utilization patterns of pitolisant. The secondary objectives were to assess clinical benefit, adherence, impact on patients' quality of life, disease burden, and patient satisfaction. We reported the results of an interim analysis after 42.6 months. Results: The population comprised 370 patients (mean age, 40 +/- 15 years; 51.4 % women; NT1, 71.4 %; NT2, 28.6 %); 364 received >= 1 dose of pitolisant. Data were available for 356 patients (97.8 %). Most patients (68.4 %) had >= 1 comorbidity (obesity [BMI >= 30], 31.9 %; neuropsychiatric, 31 %; and cardiovascular, 22.8 %). Forty-eight patients (13.2 %) had received no prior narcoleptic treatment, while 98 (31 %) were taking a previous therapy, which was switched to pitolisant. Treatment was combined with pitolisant in 218 (69 %) patients. Pitolisant was discontinued by 131 patients (35.4 %), mainly for safety reasons (14.3 %), lack of response (8.7 %), and patient decision (7.6 %). Overall, 355 treatment-emergent adverse events (3 serious) were reported by 156 patients (42.9 % of safety population), with 218 possibly treatment-related (61.4 %) in 109 patients (29.9 %). Improvements were observed in EDS, cataplexy, and quality of life. Conclusions: Pitolisant was generally safe and well tolerated in patients with NT1 and NT2 and can be used in both types. Improvements were found in EDS, cataplexy, and quality of life, with good adherence and satisfaction.

引用

页码：20 / 30

页数：11

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